🔬 This Week in Gene & Cell Therapy: Key Breakthroughs in Gene Therapy, Autoimmune Disease, and Neurology | Global Trials & Milestones 🧬
From China’s first gene therapy approval for Hemophilia B to AI-powered longevity programs and CAR-T innovations in lupus and GI cancers — this week’s update delivers powerful shifts across gene and cell therapy, neurology, autoimmune diseases, and oncology.
🚀 This Week’s Global Highlights Include:
🧬 China approves BBM-H901 gene therapy for Hemophilia B — reducing bleeding episodes by over 90 percent
🧫 CK0801 earns Orphan Drug Status, delivering long-term transfusion independence in aplastic anemia
💉 VERVE-102 achieves up to 69 percent LDL-C reduction in early CRISPR gene-editing trial
🧠 BrainStorm prepares Phase 3b ALS trial under FDA Special Protocol Assessment
💪 Descartes-08 provides 12-month relief in myasthenia gravis after a single outpatient dose
🧪 UK grants GMP license for synthetic DNA supporting Pompe and Friedreich’s ataxia trials
🧬 FT819 CAR-T earns RMAT status for lupus with signs of early remission and no major toxicities
🤖 Genflow leverages AI to target SIRT6 in aging-related gene therapy
🧫 FDA filing submitted for hiPSC-based ActiCells platform by Applied StemCell
🧠 GI cancer-focused CAR T trials show cautious progress despite biological hurdles
🧬 PRAME-targeted TCR therapy IMA203 shows over 50 percent tumor response in solid tumors
🏆 King Faisal Prize honors breakthroughs in CAR T-cell therapy and nanotech innovation
Whether you’re in clinical development, biotech strategy, or translational research, these updates highlight where the future of precision medicine is heading.
📢 Stay Ahead in Gene and Cell Therapy Research!
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